PB1900: COMBINED TREATMENT WITH VENETOCLAX PLUS HYPOMETHYLATING AGENT OR CYTARABINE VERSUS TREATMENT WITH HYPOMETHYLATING AGENT ALONE IN ACUTE MYELOID LEUKEMIA IN POOR PROGNOSIS PATIENTS.
Authors: Minerva Montalvo Saavedra, Alvaro Bienert Garcia, Belén Pérez-Pinilla, Paula Reyes González-Casanova, Marta Díaz-Lopez, Jose Gregorio Torres Quiroz, Alberto Martinez Llorens, Cielo de Los Ángeles Lorena Borja Goyeneche, Carolina Lacalzada Higueras, Bernardo Javier González González, Miguel Teodoro Hernandez Garcia, Sunil Lakhwani Lakhwani
Journal: HemaSphere (2023)

Topic: 4. Acute myeloid leukemia - Clinical Background: Venetoclax (VEN) is a selective anti-BCL2 drug which is known to have activity against several haematological malignancies. In recent years it has consistently shown its ability in the treatment of acute myeloid leukemia (AML) specially when it is combined with hypomethylating agents (HMA) or cytarabine with excellent results. Aims: Analyze and compare the efficacy and security of AML patients treated with VEN plus HMA or cytarabine versus patients treated with HMA in monotherapy at one single center. Methods: Retrospective observational longitudinal study was conducted. AML patients ineligible for intensive treatment diagnosed and treated in our center between November 2019 and January 2023 were included. Medical records of these patients were reviewed, and several clinical and biological variables were recorded. Complete remission (CR) was considered according to the International Working Group revised recommendations. Overall survival (OS) and progression free survival (PFS) were counted since the time form start treatment. Results: A total of 16 patients with diagnosis of AML were included, of which 9 patients (56,25%) received VEN plus HMA or plus cytarabine and 7 (43,75%) received only HMA, which was in all those seven cases Azacitidine (AZA). In the first group we gathered 3 cases treated with VEN plus cytarabine and 6 cases treated with VEN plus HMA. Five out of nine (55,55%) patients who received VEN and five out of seven (71,42%) patients with AZA alone were patients with secondary AML to myelodysplastic syndrome. All the patients with secondary AML received AZA previously. Among the 16 patients included, 9 of them were women and 7 male. The average age in both groups were similar: 76 years old in the VEN group and 76,5 in the AZA in monotherapy group. With the available data, ten out of sixteen patiens had adverse cytogenetic risk according to ELN2022 criteria, five in the VEN group and five in the AZA alone group. Moreover, one had favorable prognosis (AZA alone group), two had intermediate prognosis (VEN group) and three had indeterminate prognosis (both groups). In VEN group 6/9 patients (66,7%) achieved CR (including CRi) while in AZA alone group none of the patients 0/9 (0%) achieved CR, being this difference statistically significant (p=0,011). All the six patients who achieved CR (all in the VEN group) they did it after the second cycle of treatment and two of them are still on treatment without progression. There were also statistically significant differences between both groups for PFS and OS. Median PFS in VEN group was 95 days versus only 40 days in the AZA alone group (p=0,042). Median OS was 162 days for VEN group versus only 40 days for AZA alone group (p=0,025) as we can see in Figure 1. All the sixteen patients had severe neutropenia (<500/mm3) and fourteen patients had severe thrombocytopenia (<20.000/mm3) of whom 7/9 (77,7%) patients in the VEN group and 7/7 patients (100%) in the AZA alone group (difference not significant). Fifteen patients had febrile neutropenia, 9/9 (100%) in VEN group and 6/7 (85,7%) in AZA alone group (difference not significant). Summary/Conclusion: Our collected data in real life show an excellent activity and good response ratio of VEN associated with HMA or cytarabine in a cohort of AML patients with mostly poor prognosis with previous treatment. Patients who received VEN plus HMA or cytarabine benefit from a longer PFS and OS than patients treated with HMA alone.Keywords: Hypomethylating agents, Therapy-related AML, Venetoclax, AML